Use of human growth hormone in multiple system atrophy

Abstract

The invention relates to the use of a substance, which binds to and initiates signaling of the human growth hormone (hGH) receptor or a substance, which stimulates release or potentiates the activity of endogenous hGH, for treatment and / or prevention of Parkinsonism-Plus Syndromes. In particular, the invention relates to the use of hGH for the treatment and / or prevention of Multiple System Atrophy.

Description

FIELD OF INVENTION [0001] The present invention is in the field of neurologic diseases. More specifically, it relates to the use of human growth hormone for the manufacture of a medicament for treatment and / or prevention of Parkinsonism-Plus Syndromes, and in particular Multiple System Atrophy (MSA). BACKGROUND OF THE INVENTION [0002] Human growth hormone (hGH), also known as somatropin (INN) or somatotropin, is a protein hormone produced and secreted by the somatotropic cells of the anterior pituitary. Human growth hormone plays a key role in somatic growth in childhood and in metabolism in adulthood through its effects on the metabolism of proteins, carbohydrates and lipids. [0003] Human growth hormone is a single polypeptide chain of 191 amino acids (Bewly et al, 1972) having two disulfide bonds, one between Cys-53 and Cys-165, forming a large loop in the molecule, and the other between Cys-182 and Cys-189, forming a small loop near the C-terminus. The DNA sequence that confirmed...

AI Technical Summary

Benefits of technology

The present invention is based on the discovery that human growth hormone can benefit patients with a disease called Parkinsonism-Plus Syndromes, specifically Multiple System Atrophy. The invention relates to using substances that bind to and activate the human growth hormone receptor, or substances that stimulate the release or activity of endogenous human growth hormone, for the treatment and prevention of Parkinsonism-Plus Syndromes, including Multiple System Atrophy. The invention also includes the use of nucleic acid molecules, vectors, and genetically modified cells to produce these substances for treatment and prevention purposes. The technical effect of the invention is the development of new treatments for Parkinsonism-Plus Syndromes, particularly Multiple System Atrophy, which is currently untreatable.

Problems solved by technology

Asparagine and glutamine residues in proteins are susceptible to deamidation reactions under appropriate conditions.

Methionine residues in proteins are susceptible to oxidation, primarily to the sulfoxide.

Complaints in these patients have mainly been of tiredness, low energy and lack of initiative, lack of concentration, memory difficulties and irritability.

The patient has difficulty focusing and meeting the eye of the clinician and has a staring gaze due to the involuntary persistence of ocular fixation.

Severe postural instability occurs most prominently in the middle of the course of the disease and may be a dominant problem.

Multiple traumas with fractures of the arms and legs, even lethal trauma due to falling may occur.

CBD is a rare condition and reliable prevalence data are not available.

The asymmetry of symptoms is, however, persistent and the risk of confusing the syndrome with PSP is therefore small.

However, the clinical differentiation between these disorders is difficult.

Orthostatic hypotension is common and may cause dizziness, dimming of vision, head or neck pain, yawning, temporary confusion, slurred speech and if the hypotension is severe the patient may faint upon arising from a recumbent position.

The nomenclature of the different manifestations of MSA has been variable and has probably delayed the awareness of the disease.

The prognosis becomes poor when falls in blood pressure with repeated syncope has developed.

This test is a complex whole-body motor task that requires that the patient is able to walk unaided.